WO2017190214A1 - 4hpr and its use in the culturing of hematopoietic stem cells - Google Patents

4hpr and its use in the culturing of hematopoietic stem cells Download PDF

Info

Publication number
WO2017190214A1
WO2017190214A1 PCT/CA2017/000107 CA2017000107W WO2017190214A1 WO 2017190214 A1 WO2017190214 A1 WO 2017190214A1 CA 2017000107 W CA2017000107 W CA 2017000107W WO 2017190214 A1 WO2017190214 A1 WO 2017190214A1
Authority
WO
WIPO (PCT)
Prior art keywords
4hpr
cells
hsc
expansion
population
Prior art date
Application number
PCT/CA2017/000107
Other languages
English (en)
French (fr)
Inventor
John Dick
Stephanie XIE
Elisa Laurenti
Laura Garcia PRAT
Robin Thomas FERRARI
Original Assignee
University Health Network
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University Health Network filed Critical University Health Network
Priority to EP17792305.9A priority Critical patent/EP3452581B1/de
Priority to US16/098,628 priority patent/US20190136188A1/en
Publication of WO2017190214A1 publication Critical patent/WO2017190214A1/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01N—PRESERVATION OF BODIES OF HUMANS OR ANIMALS OR PLANTS OR PARTS THEREOF; BIOCIDES, e.g. AS DISINFECTANTS, AS PESTICIDES OR AS HERBICIDES; PEST REPELLANTS OR ATTRACTANTS; PLANT GROWTH REGULATORS
    • A01N1/00—Preservation of bodies of humans or animals, or parts thereof
    • A01N1/02—Preservation of living parts
    • A01N1/0205—Chemical aspects
    • A01N1/021—Preservation or perfusion media, liquids, solids or gases used in the preservation of cells, tissue, organs or bodily fluids
    • A01N1/0226—Physiologically active agents, i.e. substances affecting physiological processes of cells and tissue to be preserved, e.g. anti-oxidants or nutrients
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0018—Culture media for cell or tissue culture
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2500/00—Specific components of cell culture medium
    • C12N2500/30—Organic components
    • C12N2500/38—Vitamins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/30—Hormones
    • C12N2501/38—Hormones with nuclear receptors
    • C12N2501/385—Hormones with nuclear receptors of the family of the retinoic acid recptor, e.g. RAR, RXR; Peroxisome proliferator-activated receptor [PPAR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/999—Small molecules not provided for elsewhere
PCT/CA2017/000107 2016-05-03 2017-05-03 4hpr and its use in the culturing of hematopoietic stem cells WO2017190214A1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP17792305.9A EP3452581B1 (de) 2016-05-03 2017-05-03 4hpr und dessen verwendung in der züchtung von hämatopoetischen stammzellen
US16/098,628 US20190136188A1 (en) 2016-05-03 2017-05-03 4hpr and its use in the culturing of hematopoietic stem cells

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662331019P 2016-05-03 2016-05-03
US62/331,019 2016-05-03

Publications (1)

Publication Number Publication Date
WO2017190214A1 true WO2017190214A1 (en) 2017-11-09

Family

ID=60202572

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/CA2017/000107 WO2017190214A1 (en) 2016-05-03 2017-05-03 4hpr and its use in the culturing of hematopoietic stem cells

Country Status (3)

Country Link
US (1) US20190136188A1 (de)
EP (1) EP3452581B1 (de)
WO (1) WO2017190214A1 (de)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021148720A1 (en) * 2020-01-24 2021-07-29 Faron Pharmaceuticals Oy Method for promoting expansion of hematopoietic stem cells and agent for use in the method

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8088822B2 (en) * 2008-07-08 2012-01-03 Ji Zhang Use of fenretinide or bioactive derivatives thereof and pharmaceutical compositions comprising the same

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL152904A0 (en) * 2002-01-24 2003-06-24 Gamida Cell Ltd Utilization of retinoid and vitamin d receptor antagonists for expansion of renewable stem cell populations
WO2007033331A2 (en) * 2005-09-14 2007-03-22 Duke University Stem cells
CN101623277B (zh) * 2008-07-08 2014-07-16 上海交通大学医学院附属瑞金医院 芬维a胺及其活性衍生物的新用途及其药物组合物

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8088822B2 (en) * 2008-07-08 2012-01-03 Ji Zhang Use of fenretinide or bioactive derivatives thereof and pharmaceutical compositions comprising the same

Non-Patent Citations (44)

* Cited by examiner, † Cited by third party
Title
ANTHONY E. BOITANO ET AL.: "Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells", SCIENCE, vol. 329, no. 5997, 5 August 2010 (2010-08-05), pages 1345 - 1348, XP055558705, DOI: 10.1126/science.1191536
BENVENISTE PCANTIN CHYAM DISCOVE NN: "Hematopoietic stem cells engraft in mice with absolute efficiency", NAT IMMUNOL, vol. 4, 2003, pages 708 - 13
BENVENISTE PFRELIN CJANMOHAMED SBARBARA MHERRINGTON RHYAM DISCOVE NN: "Intermediate-term hematopoietic stem cells with extended but time-limited reconstitution potential", CELL STEM CELL, vol. 6, 2010, pages 48 - 58
BOITANO AEWANG JROMEO RBOUCHEZ LCPARKER AESUTTON SEWALKER JRFLAVENY CAPERDEW GHDENISON MS: "Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells", SCIENCE, vol. 329, 2010, pages 1345 - 8, XP055558705, DOI: 10.1126/science.1191536
CHANDA BDITADI AISCOVE NNKELLER G: "Retinoic acid signaling is essential for embryonic hematopoietic stem cell development", CELL, vol. 155, 2013, pages 215 - 27, XP028729738, DOI: 10.1016/j.cell.2013.08.055
CSASZAR EKIROUAC DCYU MWANG WQIAO WCOOKE MPBOITANO AEITO CZANDSTRA PW: "Rapid expansion of human hematopoietic stem cells by automated control of inhibitory feedback signaling", CELL STEM CELL, vol. 10, 2012, pages 218 - 29, XP028397787, DOI: 10.1016/j.stem.2012.01.003
DELANEY C: "Expansion of Human Hematopoietic Stem Cells", THOMAS' HEMATOPOIETIC CELL TRANSPLANTATION, vol. 2016, pages 62 - 70
DOULATOV SNOTTA FLAURENTI EDICK JE: "Hematopoiesis: a human perspective", CELL STEM CELL, vol. 10, 2012, pages 120 - 36, XP028397783, DOI: 10.1016/j.stem.2012.01.006
EPPERT KTAKENAKA KLECHMAN ERWALDRON LNILSSON BVAN GALEN PMETZELER KHPOEPPL ALING VBEYENE J: "Stem cell gene expression programs influence clinical outcome in human leukemia", NAT MED, vol. 17, 2011, pages 1086 - 93, XP055611746, DOI: 10.1038/nm.2415
FARES ICHAGRAOUI JGAREAU YGINGRAS SRUEL RMAYOTTE NCSASZAR EKNAPP DJMILLER PNGOM M: "Cord blood expansion. Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal", SCIENCE, vol. 345, 2014, pages 1509 - 12
GARCIA-PRAT LMARTINEZ-VICENTE MPERDIGUERO EORTET LRODRIGUEZ-UBREVA JREBOLLO ERUIZ-BONILLA VGUTARRA SBALLESTAR ESERRANO AL: "Autophagy maintains sternness by preventing senescence", NATURE, vol. 529, 2016, pages 37 - 42
GOESSLING WALLEN RSGUAN XJIN PUCHIDA NDOVEY MHARRIS JMMETZGER MEBONIFACINO ACSTRONCEK D: "Prostaglandin E2 enhances human cord blood stem cell xenotransplants and shows long-term safety in preclinical nonhuman primate transplant models", CELL STEM CELL, vol. 8, 2011, pages 445 - 58, XP055722086, DOI: 10.1016/j.stem.2011.02.003
HO T. T. ET AL.: "Autophagy maintains the metabolism andfunction of young and old stem cells", NATURE, vol. 543, 9 March 2017 (2017-03-09), pages 205 - 210, XP055435861, ISSN: 1476-4687, Retrieved from the Internet <URL:http://palgrave.nature.com/nature/journal/v543/n7644/full/nature21388.html> *
HO TTWARR MRADELMAN ERLANSINGER OMFLACH JVEROVSKAYA EVFIGUEROA MEPASSEGUE E: "Autophagy maintains the metabolism and function of young and old stem cells", NATURE, vol. 543, 2017, pages 205 - 10, XP055435861, DOI: 10.1038/nature21388
IMAN FARES ET AL.: "Cord blood expansion. Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal", SCIENCE (NEW YORK, N.Y., vol. 345, no. 6203, 19 September 2014 (2014-09-19), pages 1509 - 1512
KOBERLIN MSSNIJDER BHEINZ LXBAUMANN CLFAUSTER AVLADIMER GIGAVIN ACSUPERTI-FURGA G: "A Conserved Circular Network of Coregulated Lipids Modulates Innate Immune Responses", CELL, vol. 162, 2015, pages 170 - 83
LAI W-LWONG N-S: "The PERK/eIF2a signaling pathway of Unfolded Protein Response is essential for N-(4-hydroxyphenyl)retinamide (4HPR)-induced cytotoxicity in cancer cells", EXPERIMENTAL CELL RESEARCH, vol. 314, 2008, pages 1667 - 82, XP022627448, DOI: 10.1016/j.yexcr.2008.02.002
LAURENTI EDOULATOV SZANDI SPLUMB ICHEN JAPRIL CFAN J-BDICK JE: "The transcriptional architecture of early human hematopoiesis identifies multilevel control of lymphoid commitment", NATURE IMMUNOLOGY, vol. 14, 2013, pages 756 - 63
LAURENTI EFRELIN CXIE SFERRARI RDUNANT CFZANDI SNEUMANN APLUMB IDOULATOV SCHEN J: "CDK6 levels regulate quiescence exit in human hematopoietic stem cells", CELL STEM CELL, vol. 16, 2015, pages 302 - 13
LECHMAN ERGENTNER BNG SWSCHOOF EMVAN GALEN PKENNEDY JANUCERA SCICERI FKAUFMANN KBTAKAYAMA N: "miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells", CANCER CELL, vol. 29, 2016, pages 214 - 28, XP029412950, DOI: 10.1016/j.ccell.2015.12.011
LECHMAN ERGENTNER BVAN GALEN PGIUSTACCHINI ASAINI MBOCCALATTE FEHIRAMATSU HRESTUCCIA UBACHI AVOISIN V: "Attenuation of miR-126 activity expands HSC in vivo without exhaustion", CELL STEM CELL, vol. 1, no. 1, 2012, pages 799 - 811
LI ET AL., CELL DEATH AND DISEASE, 2014
LI YLI SQIN XHOU WDONG HYAO LXIONG L: "The pleiotropic roles of sphingolipid signaling in autophagy", CELL DEATH DIS, vol. 5, 2014, pages e1245
LIU XWSU YZHU HCAO JDING WJZHAO YCHE QJYANG B: "HIF-1alpha-dependent autophagy protects HeLa cells from fenretinide (4-HPR)-induced apoptosis in hypoxia", PHARMACOL RES, vol. 62, 2010, pages 416 - 25, XP027320681
LUND TCBOITANO AEDELANEY CSSHPALL EJWAGNER JE: "Advances in umbilical cord blood manipulation [mdash] from niche to bedside", NATURE REVIEWS CLINICAL ONCOLOGY, vol. 12, 2015, pages 163 - 74
LUO YLI LZOU PWANG JSHAO LZHOU DLIU L: "Rapamycin enhances long-term hematopoietic reconstitution of ex vivo expanded mouse hematopoietic stem cells by inhibiting senescence", TRANSPLANTATION, vol. 97, 2014, pages 20 - 9, XP055562585, DOI: 10.1097/TP.0b013e3182a7fcf8
MACEYKA MSPIEGEL S: "Sphingolipid metabolites in inflammatory disease", NATURE, vol. 510, 2014, pages 58 - 67, XP055689592, DOI: 10.1038/nature13475
MATHEW RKARP CMBEAUDOIN BVUONG NCHEN GCHEN HYBRAY KREDDY ABHANOT GGELINAS C, CELL, vol. 137, 2009, pages 1062 - 75
MERRILL AH, JR: "Sphingolipid and glycosphingolipid metabolic pathways in the era of sphingolipidomics", CHEM REV, vol. 111, 2011, pages 6387 - 422
NOTTA FDOULATOV SLAURENTI EPOEPPL AJURISICA IDICK JE: "Isolation of single human hematopoietic stem cells capable of long-term multilineage engraftment", SCIENCE, vol. 333, 2011, pages 218 - 21, XP055069664, DOI: 10.1126/science.1201219
NOTTA FZANDI STAKAYAMA NDOBSON SGAN 01WILSON GKAUFMANN KBMCLEOD JLAURENTI EDUNANT CF: "Distinct routes of lineage development reshape the human blood hierarchy across ontogeny", SCIENCE, vol. 351, 2016, pages aab2116
OUIMET MFRANKLIN VMAK ELIAO XTABAS IMARCEL YL: "Autophagy regulates cholesterol efflux from macrophage foam cells via lysosomal acid lipase", CELL METAB, vol. 13, 2011, pages 655 - 67, XP028376152, DOI: 10.1016/j.cmet.2011.03.023
PARK, B. ET AL.: "Hematopoietic stem cell expansion and generation: the ways to make a breakthrough", BLOOD RESEARCH, vol. 50, no. 4, December 2015 (2015-12-01), pages 194 - 203, XP055435854, ISSN: 2288-0011, Retrieved from the Internet <URL:http://dx.doi.Org/10.5045/br.2015.50.4.194> [retrieved on 20170814] *
RAHMANIYAN MCURLEY RWOBEID LMHANNUN YAKRAVEKA JM: "Identification of Dihydroceramide Desaturase as a Direct in Vitro Target for Fenretinide", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 286, 2011, pages 24754 - 64
RAHMANLYAN ET AL., JBC, 2011
See also references of EP3452581A4
SIDDIQUE MMLI YWANG LCHING JMAL MILKAYEVA OWU YJBAY BHSUMMERS SA: "Ablation of dihydroceramide desaturase 1, a therapeutic target for the treatment of metabolic diseases, simultaneously stimulates anabolic and catabolic signaling", MOL CELL BIOL, vol. 33, 2013, pages 2353 - 69
SIDDIQUE, MCB, 2013
SINGH RKAUSHIK SWANG YXIANG YNOVAK IKOMATSU MTANAKA KCUERVO AMCZAJA MJ: "Autophagy regulates lipid metabolism", NATURE, vol. 458, 2009, pages 1131 - 5, XP055157034, DOI: 10.1038/nature07976
TANIGUCHI MKITATANI KKONDO THASHIMOTO-NISHIMURA MASANO SHAYASHI AMITSUTAKE SIGARASHI YUMEHARA HTAKEYA H: "Regulation of autophagy and its associated cell death by ''sphingolipid rheostat'': reciprocal role of ceramide and sphingosine 1-phosphate in the mammalian target of rapamycin pathway", J BIOL CHEM, vol. 287, 2012, pages 39898 - 910
VAN GALEN PKRESO AMBONG NKENT DGFITZMAURICE TCHAMBERS JEXIE SLAURENTI EHERMANS KEPPERT K: "The unfolded protein response governs integrity of the haematopoietic stem-cell pool during stress", NATURE, vol. 510, 2014, pages 268 - 72
VAN GALEN PKRESO AWIENHOLDS ELAURENTI EEPPERT KLECHMAN ERMBONG NHERMANS KDOBSON SAPRIL C: "Reduced lymphoid lineage priming promotes human hematopoietic stem cell expansion", CELL STEM CELL, vol. 14, 2014, pages 94 - 106
WAGNER JEBRUNSTEIN CGBOITANO AEDEFOR TEMCKENNA DSUMSTAD DBLAZAR BRTOLAR JLE CJONES J: "Phase / Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft", CELL STEM CELL, vol. 18, 2016, pages 144 - 55, XP055558717, DOI: 10.1016/j.stem.2015.10.004
WARR MRBINNEWIES MFLACH JREYNAUD DGARG TMALHOTRA RDEBNATH JPASSEGUE E: "FOX03A directs a protective autophagy program in haematopoietic stem cells", NATURE, 2013

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021148720A1 (en) * 2020-01-24 2021-07-29 Faron Pharmaceuticals Oy Method for promoting expansion of hematopoietic stem cells and agent for use in the method

Also Published As

Publication number Publication date
EP3452581A1 (de) 2019-03-13
EP3452581A4 (de) 2019-12-04
US20190136188A1 (en) 2019-05-09
EP3452581B1 (de) 2023-01-11

Similar Documents

Publication Publication Date Title
Tower Stress and stem cells
Perry et al. Cooperation between both Wnt/β-catenin and PTEN/PI3K/Akt signaling promotes primitive hematopoietic stem cell self-renewal and expansion
Gonzalez et al. Neural stem cells derived from human parthenogenetic stem cells engraft and promote recovery in a nonhuman primate model of Parkinson's disease
Yoshihara et al. Thrombopoietin/MPL signaling regulates hematopoietic stem cell quiescence and interaction with the osteoblastic niche
Lauridsen et al. Differences in cell cycle status underlie transcriptional heterogeneity in the HSC compartment
Newell et al. Mesenchymal stem cells shift mitochondrial dynamics and enhance oxidative phosphorylation in recipient cells
US20150164952A1 (en) Methods of Ex Vivo Expansion of Blood Progenitor Cells, and Generation of Composite Grafts
Cao et al. Autophagy regulates the cell cycle of murine HSPCs in a nutrient-dependent manner
US20210322485A1 (en) Mitochondrial augmentation therapy with stem cells enriched with functional mitochondria
US20150246075A1 (en) Methods for enhancing hematopoietic stem/progenitor cell engraftment
Zhang et al. Acute myeloid leukemia cells educate mesenchymal stromal cells toward an adipogenic differentiation propensity with leukemia promotion capabilities
EP3851112A1 (de) Transplantation von mitochondrien in lymphorgan und zusammensetzung dafür
Du et al. Cited2 is required for the maintenance of glycolytic metabolism in adult hematopoietic stem cells
US20210386787A1 (en) Hematopoietic Stem and Progenitor Cell Expansion System
Ramkumar et al. Smurf2 regulates hematopoietic stem cell self‐renewal and aging
EP3452581B1 (de) 4hpr und dessen verwendung in der züchtung von hämatopoetischen stammzellen
US20170333483A1 (en) Methods for enhancing hematopoietic stem/progenitor cell engraftment
Li et al. Human umbilical cord mesenchymal stem cell‑derived exosomes improve ovarian function in natural aging by inhibiting apoptosis
Fu et al. Vitrification of rhesus macaque mesenchymal stem cells and the effects on global gene expression
Lu Sleeping beauty wakes up the clonal succession model for homeostatic hematopoiesis
US20190269705A1 (en) Methods and materials for treating graft versus host disease
US20190000885A1 (en) Treatment with angiogenin to enhance hematopoietic reconstitution
Stolzenbach et al. Non-neutral clonal selection and its potential role in mammalian germline stem cell dysfunction with advancing age
Balcerek The Role of Lnk Adaptor Protein in Hematopoietic Stem Cell Genome Stability and Self-Renewal
Nguyen-McCarty Characterizing a Signaling Network That Maintains Hematopoietic Stem Cells

Legal Events

Date Code Title Description
NENP Non-entry into the national phase

Ref country code: DE

121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 17792305

Country of ref document: EP

Kind code of ref document: A1

ENP Entry into the national phase

Ref document number: 2017792305

Country of ref document: EP

Effective date: 20181203